AI Summary
This article reports on a study conducted by the Children's Oncology Group focusing on patients with embryonal sarcoma of the liver (ESL), a rare mesenchymal tumor most commonly found in childhood. The study evaluated the clinical features and outcomes of young patients with ESL who were treated with a risk-based strategy involving chemotherapy, surgery, and optional radiotherapy. Results showed that delaying tumor resection until after chemotherapy decreased the risk of intraoperative tumor rupture and improved the chances of achieving adequate surgical margins. The overall 5-year survival rate was found to be 95%, with a positive correlation between positive surgical margins and local recurrence risk. This study demonstrates that a treatment approach involving neoadjuvant chemotherapy can lead to favorable outcomes for pediatric and young adult patients with ESL.
Abstract
Background
Embryonal sarcoma of the liver (ESL) is a rare mesenchymal tumor most common in childhood; the optimal treatment approach is uncertain. The clinical features and outcomes of patients with ESL enrolled in a Children’s Oncology Group (COG) clinical trial that evaluated a risk-based strategy for treating soft tissue sarcomas in patients aged <30 years were evaluated.
Methods
This subset analysis included patients with ESL enrolled in COG study ARST0332. Central review of records, pathology, and imaging confirmed the diagnosis, presenting features, and surgery extent and complications. All patients received dose-intensive ifosfamide/doxorubicin chemotherapy, with cycle timing dependent on surgery and radiotherapy. Tumor resection occurred before study entry or after four cycles of chemotherapy; radiotherapy for residual tumor was optional.
Results
Thirty-nine eligible/evaluable patients with ESL were analyzed. All tumors were >10 cm in diameter; four were metastatic. Tumor resection was performed upfront in 23 and delayed in 16. Positive surgical margins (n = 6) and intraoperative tumor rupture (n = 6) occurred only in upfront resections. Eight patients received radiotherapy. Estimated 5-year event-free and overall survival were 79% (95% confidence interval [CI], 65%–93%) and 95% (95% CI, 87%–100%), respectively. Positive margins increased the local recurrence risk. One of 13 patients with documented hemorrhagic ascites and/or tumor rupture developed extrahepatic intra-abdominal tumor recurrence.
Conclusions
The treatment strategy used in ARST0332 achieved favorable outcomes for patients with ESL despite a substantial proportion having high-risk disease features. Deferring tumor resection until after neoadjuvant chemotherapy may decrease the risk of intraoperative tumor rupture and improve the likelihood of adequate surgical margins.